Core Viewpoint - Mesoblast Limited has provided an update on Ryoncil (remestemcel-L-rknd), the first FDA-approved mesenchymal stromal cell product for treating steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children aged 2 months and older, highlighting its early survival rates and ongoing commercial efforts to expand its use [1][8]. Group 1: Clinical Outcomes - Among the first 25 patients treated with Ryoncil, 21 patients (84%) survived and completed the initial 28-day treatment regimen as per FDA approval [2] - The four patients who did not complete the treatment had previously failed other therapies and died from severe SR-aGvHD within 28 days, underscoring the importance of early intervention [2] Group 2: Patient Access and Coverage - Mesoblast has established a patient access hub, MyMesoblast™, to facilitate ordering Ryoncil, ensuring that patients can access this therapy [3] - Ryoncil is covered by government and commercial payers, reaching over 260 million U.S. lives, with specific billing codes issued by CMS enhancing its usage under federal coverage [4] Group 3: Market Expansion and Future Trials - The company aims to expand Ryoncil's FDA label to include adults with severe SR-aGvHD, targeting a market size approximately three times larger than the pediatric population [5] - A pivotal trial for Ryoncil in adults is set to commence site enrollment this quarter, in collaboration with the NIH-funded Bone Marrow Transplant Clinical Trials Network [5] Group 4: Company Overview and Technology - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [7] - The company is also developing additional therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [9]

Core Insights - Mesoblast Limited announced that remestemcel-L demonstrated superior efficacy and safety compared to ruxolitinib for treating steroid-refractory acute graft versus host disease (SR-aGvHD) at the 67th ASH Annual meeting [1][2] Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory diseases, utilizing a proprietary mesenchymal lineage cell therapy technology platform [4] - The company has a strong intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection until at least 2044 in major markets [7] - Mesoblast's manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8] Product Information - Ryoncil (remestemcel-L) is the first FDA-approved mesenchymal stromal cell therapy for pediatric patients aged 2 months and older with SR-aGvHD [5] - The therapy is being developed for additional indications, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease, as well as heart failure and chronic low back pain [6] Clinical Study Findings - The meta-analysis included 2,732 patients across 11 studies, with remestemcel-L showing superior outcomes in complete and overall remission compared to ruxolitinib [2] - Both therapies improved quality of life, but remestemcel-L had better results in terms of hematology, cardiac, and hepatic adverse events [2]

Core Insights - Mesoblast Limited reported significant revenue growth driven by increased adoption of its product Ryoncil, with a permanent J-Code enhancing billing and reimbursement processes [2][4]. Financial Highlights - Revenue from cell therapy products reached US$20.6 million for the quarter ended September 30, 2025, up from US$12.9 million in the previous quarter, and over ten times greater than the same quarter last year [4]. - Ryoncil gross sales increased by 66% to US$21.9 million, while net sales rose by 69% to US$19.1 million after a 12.7% gross to net adjustment [4]. - The company reported a net operating cash spend of US$14.9 million, a reduction of US$1.7 million compared to the previous quarter, and had US$145 million cash on hand as of September 30, 2025 [4]. Operational Highlights - Mesoblast has onboarded 40 transplant centers since the product launch and identified 45 priority transplant centers that account for approximately 80% of U.S. pediatric transplants [4]. - Over 260 million U.S. lives are insured by commercial and government payers for Ryoncil, with federal Medicaid coverage in place [4]. - The company established a patient access hub, MyMesoblast™, to assist patients with insurance coverage and access to Ryoncil [4]. Regulatory and Development Updates - A specific J-Code (J3402) was assigned to Ryoncil by CMS, effective October 1, 2025, facilitating easier billing and reimbursement [4]. - Mesoblast plans to conduct a pivotal trial for Ryoncil in adults with severe steroid-refractory acute graft-versus-host disease (SR-aGvHD), aiming to extend its label from children to adults [5].